RTI Health Solutions, Research Triangle Park, NC 27709-2194, USA.
Pharmacoeconomics. 2011 Nov;29(11):977-88. doi: 10.2165/11593370-000000000-00000.
Angiogenesis inhibitor agents have been shown to be effective in increasing progression-free survival (PFS) in patients with renal cell carcinoma (RCC); however, these treatments have different toxicity profiles.
Our objective was to quantify patients' benefit-risk preferences for RCC treatments and relative importance of attributes in a common metric.
US residents aged ≥18 years with RCC completed a web-enabled, choice-format conjoint survey that presented a series of 12 trade-off questions, each including a pair of hypothetical RCC treatment profiles. Each profile was defined by efficacy (PFS, when overall survival held constant), tolerability effects (fatigue/tiredness, diarrhoea, hand-foot syndrome [HFS], mouth sores) and serious adverse events (liver failure, blood clot). Trade-off questions were based on a predetermined experimental design with known statistical properties. Random-parameters logit was used to analyse the data.
A total of 138 patients completed the survey. PFS was the most important attribute for patients over the range of levels included in the survey, while remaining attributes were ranked in decreasing order of importance: fatigue/tiredness, diarrhoea, liver failure, HFS, blood clot and mouth sores. In order to increase PFS by 11 months, patients would be willing to accept a maximum level of absolute blood clot risk of 3.1% (95% CI 1.5, 5.3) or liver failure risk of 2.0% (95% CI 1.0, 3.3).
A 22-month change in PFS was shown to be the most important improvement for patients. Severe fatigue/tiredness and diarrhoea were rated as the most troublesome tolerability effects of RCC treatment. Patients were likely willing to accept significant treatment-related risks of 2-3% for liver failure and blood clot to increase PFS by 11 months.
血管生成抑制剂已被证明可有效提高肾细胞癌（RCC）患者的无进展生存期（PFS）；然而，这些治疗方法具有不同的毒性特征。
我们的目的是量化患者对 RCC 治疗的获益-风险偏好，并在共同指标中量化各属性的相对重要性。
美国年龄≥18 岁的 RCC 患者完成了一项基于网络的、选择格式的联合调查，该调查提出了一系列 12 个权衡问题，每个问题都包括一对假设的 RCC 治疗方案。每个方案由疗效（当总生存保持不变时的 PFS）、耐受性影响（疲劳/疲倦、腹泻、手足综合征[HFS]、口腔疼痛）和严重不良事件（肝衰竭、血栓形成）定义。权衡问题基于具有已知统计特性的预定实验设计。使用随机参数对数比分析数据。
共有 138 名患者完成了调查。在调查涵盖的所有水平范围内，PFS 是患者最重要的属性，而其余属性则按重要性降序排列：疲劳/疲倦、腹泻、肝衰竭、HFS、血栓形成和口腔疼痛。为了将 PFS 提高 11 个月，患者愿意接受最大的绝对血栓形成风险为 3.1%（95%CI 1.5,5.3）或肝衰竭风险为 2.0%（95%CI 1.0,3.3）。
患者认为 PFS 改变 22 个月是最重要的改善。严重疲劳/疲倦和腹泻被评为 RCC 治疗最麻烦的耐受性影响。为了将 PFS 提高 11 个月，患者可能愿意接受 2-3%的治疗相关风险，即肝衰竭和血栓形成的风险，以提高 PFS。
